Mishaps show stem cell clinics need regulation, journal says.
An experimental stem cell transplant procedure in which a private Florida clinic performed intravitreal injections on three women resulted in blindness for one and severe vision loss for the other two. The patients were seeking treatment for non-neovascular age-related macular degeneration (AMD).
Reporting in the New England Journal of Medicine, Ajay Kuriyan, MD, (photo) and colleagues said the procedures involved using stem cells derived from patients’ adipose tissue, a technique they said is increasingly being used for a variety of treatments at commercial stem cell clinics. That includes attempts to restore vision, with 13 such clinical trials involving eye injections of various kinds of stem cells registered with the US Food and Drug Administration (FDA). Four are in the US, the authors said.
Among the controversial things done at the Florida clinic, the authors said, was to inject both of each patients’ eyes at the same time, putting their eyesight in double jeopardy. The mishaps all occurred in 2015 and were documented after patients sought treatment elsewhere.
In the journal article, the authors say the mishaps also highlight several larger problems.
Stem cell clinics are only loosely regulated in the US and the trials conducted there and listed on Clinicaltrials.gov are not necessarily endorsed by the National Institutes of Health. A spokeswoman for the NIH told a New York Times reporter that “ClinicalTrials.gov does not independently verify the scientific validity or relevance of the trial itself beyond a limited quality control review.”
As a result, these clinics—some of which are heavily advertised in consumer publications— “are treating patients with little oversight and with no proof of efficacy,” the authors wrote. The women described in the article all said they had found the clinic and the treatment by searching ClinicalTrials.gov. They all paid $5,000 each for the injections. In legitimate clinical trials, patients are not charged.
The authors said the clinic used autologous stem cells drawn from patients’ abdominal fat and mixed with their blood, which was injected into patients’ eyes. The authors questioned whether the mixture even contained stem cells. They also noted that there is only “sparse evidence” that this type of stem cell will later differentiate into the retinal pigment epithelial or photoreceptor cells that could help restore vision.
Some stem cell research—not done at the Florida clinic-- is more promising for vision problems. There is evidence however that human embryonic stem cells may be able to help when a suspension is injected under the retina. Such injections “have a good safety profile,” the authors wrote. They also noted that these cells are much harder to come by and that the commercial clinics use the fat-derived preparations “because of the ease of obtaining and preparing these cells.”
According to a lawsuit filed by one Florida patient, the injection was done by a nurse practitioner. The journal article authors did not fault the injection technique itself but said “the complications seen in the three patients described in this series are probably due to the stem-cell preparations.”
The patients’ vision loss was associated with ocular hypertension, hemorrhagic retinopathy, vitreous hemorrhage, combined traction and rhegmatogenous retinal detachment or lens dislocation. A year after the disastrous procedures, patients’ visual acuity ranged from 20/200 to no light perception.
The patients ages were 72 (the one who was blinded), 78, and 88.
According to news accounts, the clinic is part of a company called US Stem Cell, and at the time of the Florida procedures in 2015 was known as Bioheart. The company has said the clinic did not need FDA approval for its treatments since they involved the patients’ own cells and tissues. The Times quoted a company spokeswoman as saying the women were not treated as part of the clinical trial and that the planned trial was discontinued, adding that the clinic had treated thousands of patients for other conditions successfully.
The paper’s authors said they contacted the FDA after the patients came to them for treatment, but so far have not heard back from the agency as to whether it is investigating. The FDA has had no comment.
The journal article is entitled “Vision Loss after Intravitreal Injection of Autologous ‘Stem Cells’ for AMD.”